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Impact of ATP-binding cassette A3 (ABCA3) mutations on alveolar type 2 cell function and genetic complementation

Award Number: R03TR004814
ORGANIZATION: NATIONAL CENTER FOR ADVANCING TRANSLATIONAL SCIENCES
OPDIV: NIH
AWARD CLASS: DISCRETIONARY
AWARD ACTIVITY TYPE: SCIENTIFIC/HEALTH RESEARCH (INCLUDES SURVEYS)
PERIOD OF PERFORMANCE START DATE: 08/01/2024
PERIOD OF PERFORMANCE END DATE: 07/31/2025

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Impact of ATP-binding cassette A3 (ABCA3) mutations on alveolar type 2 cell function and genetic complementation - Summary/Abstract Pulmonary surfactant is comprised of phospholipids and protein components and helps prevent lung collapse by lowering surface tension in alveolar lining fluid. ATP-binding cassette A3 (ABCA3) is a cyclic AMP-activated phospholipid transporter that localizes to the limiting membrane of lamellar bodies in alveolar type 2 epithelial cells (AEC2s). Genetic variants in ABCA3 can cause severe neonatal respiratory distress syndrome (RDS) or later onset pulmonary fibrosis and are the most common genetic causes of childhood interstitial lung diseases (chILD). Other than lung transplant, there are no specific treatments for severe cases of ABCA3 deficiency. Since ABCA3 deficiency is autosomal recessive, it should be amenable to gene addition strategies. Complementing the loss of function with a full length ABCA3 cDNA reverses surfactant deficiency phenotypes in vitro. Our overall goals are to 1) understand the biological effects of ABCA3 mutations in human AEC2s and 2) actualize a gene therapy strategy to modify the disease course by efficiently and persistently expressing ABCA3 at physiological levels in AEC2s.


Issue Date FY	Funding FY	Legal Entity Name	Legal Entity Address	Legal Entity City	Legal Entity State	Legal Entity Zip Code	Legal Entity COUNTY	Legal Entity COUNTRY	Assistance Listing	Award Code	Budget Year	Action Date	Action Type	Action Amount

Issue Date FY: 2026 ( Subtotal = -$6,431 )
2026	2024	THE UNIVERSITY OF IOWA	105 JESSUP HALL	IOWA CITY	IA	52242	JOHNSON	USA	National Center for Advancing Translational Sciences	000	1	12/12/2025	NEW	-$6,431
														Subtotal = -$6,431

Issue Date FY: 2024 ( Subtotal = $173,175 )
2024	2024	THE UNIVERSITY OF IOWA	105 JESSUP HALL	IOWA CITY	IA	52242	JOHNSON	USA	National Center for Advancing Translational Sciences	000	1	7/24/2024	NEW	$173,175
														Subtotal = $173,175

Grand Total All Awards = $166,744

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Impact of ATP-binding cassette A3 (ABCA3) mutations on alveolar type 2 cell function and genetic complementation

Award Number: R03TR004814

ORGANIZATION: NATIONAL CENTER FOR ADVANCING TRANSLATIONAL SCIENCES

OPDIV: NIH

AWARD CLASS: DISCRETIONARY

AWARD ACTIVITY TYPE: SCIENTIFIC/HEALTH RESEARCH (INCLUDES SURVEYS)

PERIOD OF PERFORMANCE START DATE: 08/01/2024

PERIOD OF PERFORMANCE END DATE: 07/31/2025

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